In most Western countries, an assessment of clinical benefit is usually required before a pharmaceutical drug or other health care technology can access market and be reimbursed. In each country, pharmaceutical companies are faced with different requirements. Large heterogeneity is found with regard to the scope, procedure, methodology, and results of the health technology appraisal (HTA).
In general, a comparative analysis of clinical efficacy and/or effectiveness is performed to support national decisions regarding reimbursement or pricing. The aim is to determine whether and to which extent an intervention does more good than harm as compared with one or more alternative intervention(s) when provided under usual conditions of health care practice with the aim to achieve the desired treatment outcome. Ideally, randomised controlled trials (RCTs) that combine generalisability and high result certainty should be conducted to gather data on patient-relevant endpoints of acceptable validity. In these “pragmatic trials”, “real world trials”, or “practical trials” there are no defined study visits and no special requirements for the patients, which would go beyond what is necessary for treatment.
The benefit evaluations can be divided into rapid (or early) assessments for single new pharmaceuticals and full assessments for (all) available therapeutic options. While rapid assessments are often performed within a pre-specified timeframe, e.g. 6 months for early benefit assessments in Germany, there is usually no fixed timeframe for full (non-rapid) assessments. Otherwise, the methodological approaches for both types of assessments are similar. While the early assessment is usually performed soon after marketing authorisation, a full assessment is often conducted when the pharmaceutical has already been available on the market for several years and multiple comparators are available in the specific indication.
Efficacy / effectiveness spectrum
Abbr. RCT = randomised controlled trial. Adapted from High Level Pharmaceutical Forum Report 2008.